Study of Acthar® Gel (Acthar) for Amyotrophic Lateral Sclerosis (ALS)

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About 213 people with ALS will participate in this study. There will be locations in North and South America.

During the first part, participants will be randomly assigned to a group (like by flipping a coin). Out of every 3:

2 will get the study drug

1 will get a look-alike with no drug in it (placebo)

During the second part, everyone will get the study drug.

Participation will help doctors find out if Acthar can help or slow down the symptoms of ALS better than placebo.

Condition or disease

Intervention/treatment

Phase

Amyotrophic Lateral Sclerosis

Drug: ActharDrug: Placebo

Phase 2Phase 3

Detailed Description:

This is a multicenter, multiple dose study to examine the effect of Acthar on functional decline in adult participants with ALS. Approximately 213 participants will be enrolled.

Following a screening period of up to 28 days, participants with ALS and symptom onset (defined as first muscle weakness or dysarthria) ≤ 2 years prior to the Screening Visit will be randomized on a 2:1 basis to receive subcutaneous (SC) Acthar 0.2 mL (16 Units [U]) daily (QD) or SC matching placebo 0.2 mL QD for 36 weeks, followed by a 3-week taper.

Participants who complete the 36 week double-blind treatment period are eligible to enter an Open Label Extension phase in which all participants will receive Acthar 0.2 mL (16 U) daily.

Participants will be assigned to treatment group randomly (like flipping a coin). They will have a 2 out of 3 chance of receiving the study drug, and a 1 out of 3 chance of receiving placebo during the treatment period. All participants who continue into the extension period receive Acthar.

Participants receive one 0.2 mL subcutaneous (SC) injection (shot under the skin) of the study drug (Acthar), daily for up to 36 weeks.

Those who do not continue into the extension period will have 3 weeks of tapering off the drug, ending their participation by Week 39.

Drug: Acthar

Repository corticotropin for subcutaneous injection

Other Names:

Trade name: Acthar® Gel

Generic name: repository corticotropin injection

Placebo Comparator: Arm B: Treatment Period Placebo

Participants receive one 0.2 mL SC injection that looks like Acthar, but has no drug in it (Matching Placebo), daily for up to 36 weeks.

Those who do not continue into the extension period will have 3 weeks of simulated tapering, ending their participation by Week 39.

Drug: Placebo

Matching placebo for subcutaneous injection

Other Names:

Matching Placebo

Reference Product

Experimental: Arm C: Extension Period Acthar-Acthar

Participants who receive Acthar during the treatment period and continue into the extension period do not go through the treatment-period tapering, but receive one 0.2 mL SC injection of Acthar, daily for up to 48 weeks, followed by 3 weeks of tapering off the drug, ending their participation within about 21 months.

Drug: Acthar

Repository corticotropin for subcutaneous injection

Other Names:

Trade name: Acthar® Gel

Generic name: repository corticotropin injection

Experimental: Arm D: Extension Period Placebo-Acthar

Participants who receive Placebo during the treatment period and continue into the extension period do not go through the treatment-period simulated tapering, but receive one 0.2 mL SC injection of Acthar, daily for up to 48 weeks, followed by 3 weeks of tapering off the drug, ending their participation within about 21 months.

A trained, independent rater calls each participant (or the caregiver) to administer the questionnaire. The 12 functions are rated on a scale from 0 to 4, with a highest possible score of 48. Higher scores represent better function.

Number of participants experiencing an adverse event during the Treatment Period [ Time Frame: by the end of the treatment period (within 36 Weeks) ]

Serious adverse events, non-serious treatment-emergent adverse events, and all-cause mortality are collected until the participant no longer participates in the study (estimated about 1 year for participants leaving after the treatment period). Clinically significant changes in safety measures are recorded as adverse events.

Number of participants experiencing an adverse event by the end of the Trial [ Time Frame: by the time of database lock (within 84 weeks) ]

Serious adverse events, non-serious treatment-emergent adverse events, and all-cause mortality are collected until the participant no longer participates in the study (estimated about 1 year for participants leaving after the treatment period, and two years for participants who participate also in the open label extension). Clinically significant changes in safety measures are recorded as adverse events.

The trained investigator (or designee) administers the ALSFRS-R questionnaire in person with the participant (or caregiver). The 12 functions are rated on a scale from 0 to 4, with a highest possible score of 48. Higher scores represent better function.

Extension Period: Scores on a scale for investigator-administered ALSFRS-R [ Time Frame: at Week 84 ]

The trained investigator (or designee) administers the ALSFRS-R questionnaire in person with the participant (or caregiver). The 12 functions are rated on a scale from 0 to 4, with a highest possible score of 48. Higher scores represent better function.

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

Layout table for eligibility information

Ages Eligible for Study:

18 Years to 75 Years (Adult, Older Adult)

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

No

Criteria

Inclusion Criteria:

Is 18-75 years of age at Screening

Has ALS symptom onset within 2 years prior to Screening

Has forced vital capacity (FVC) no higher than 60% at screening

If taking riluzole, is on a stable dose for 4 weeks before Screening

Exclusion Criteria:

Has tracheostomy, diaphragm pacing, or an ongoing need for assisted ventilation of any type

Has used any medication within a time period not allowed per protocol

Has history of Type 1 or Type 2 diabetes mellitus, or any clinically significant infection